Sun Pharma Advanced Research Company to sell priority review voucher for $195 Million

SPARC enters definitive agreement to monetise rare paediatric disease PRV, unlocking capital for R&D and strategic growth

SPARC announces receipt of priority review voucher associated with Sezaby approval

A PRV is a tradable voucher granted by the FDA to encourage the development of new treatments for rare pediatric diseases

Rocket Pharma gets FDA nod for first pediatric gene therapy targeting severe LAD-I

The approval covers pediatric patients without a matched sibling donor for stem cell transplant

US House passes Bill reauthorizing FDA rare disease incentive program

FDA nod to priority review to AstraZeneca’s Baxdrostat for hard-to-control hypertension

Here's a development that aims to keep your blood pressure in check. Literally!

US District Court grants summary judgement in Favour of SPARC for Sezaby PRV

FDA's withholding of the PRV was contrary to law because no drug product containing phenobarbital sodium was 'previously approved'

Merck KGaA Q3 2025 net sales remains flat amid geopolitical uncertainty

FDA approves UCB’s Kygevvi for rare genetic mitochondrial disease TK2 deficiency

Kygevvi combines two pyrimidine nucleosides, doxecitine and doxribtimine, which act by integrating deoxycytidine and deoxythymidine into skeletal muscle mitochondrial DNA

USFDA approves first gene therapy for treatment of aromatic L-amino acid decarboxylase deficiency

Aromatic L-amino acid decarboxylase deficiency is a rare genetic disorder that affects the production of some neurotransmitters

Wacker Biotech partners with Caeregen Therapeutics on regenerative treatment for vision loss

As Caeregen’s CDMO partner for CTR-107, Wacker Biotech will produce drug substance at its site in Halle, Germany, and complete clinical trial drug product production at its site in Amsterdam, the Netherlands